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Repurposing at UCSF

Finding treatments for rare conditions like MEPAN can take years and cost millions of dollars, so our initial focus is on supporting drug repurposing research.

 

These projects examine FDA-approved drugs to identify existing medicines that can address MEPAN symptoms. Because these drugs have already been reviewed by the FDA and tested in adult patients for safety, repurposing is potentially the fastest, most cost-effective path to finding a treatment.

A team at the University of San Francisco's Department of Medical Genetics is working with Perlara on a MEPAN repurposing project, and they have identified several drugs that may help patients. Read our blog post with the latest update here.

Yeast

Project Summary

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Create models of MECR gene mutations in yeast

Drugs

Screen 1,000s of existing FDA-approved drugs

Submit to FDA for clinical trial 

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Test promising drug candidates in patient skin cells

 
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